Bias, precision and statistical power of analysis of covariance in the analysis of randomized trials with baseline imbalance: A simulation study

BMC Medical Research Methodology

Volumn 14, Issue 1, 2014, Pages

  Egbewale, Bolaji E ^a   Lewis, Martyn ^a   Sim, Julius ^a  

^a KEELE UNIVERSITY   (United Kingdom)

Author keywords

Baseline imbalance; Bias; Precision; Randomized controlled trials; Statistical analysis; Statistical power

Indexed keywords

ANALYSIS OF VARIANCE; ARTICLE; COMPUTER SIMULATION; HUMAN; METHODOLOGY; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICAL ANALYSIS; STATISTICAL BIAS; STATISTICAL MODEL;

ANALYSIS OF VARIANCE; BIAS (EPIDEMIOLOGY); COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN;

EID: 84899476487     PISSN: None     EISSN: 14712288     Source Type: Journal    
DOI: 10.1186/1471-2288-14-49     Document Type: Article

References (46)

1. Rosenberger WF, Lachin JM, Randomization in Clinical Trials: Theory and Practice New York, NY: Wiley-Interscience 2002
2. Baseline imbalance in randomised controlled trials. Roberts C, Torgerson DJ, BMJ 1999 319 185 10406763
3. Baseline comparisons in randomized clinical trials. Altman DG, Doré CJ, Stat Med 1991 10 797 799 2068432
4. Adjustment of treatment effect for covariates in clinical trials: statistical and regulatory issues. Tu D, Shalay K, Pater J, Drug Inf J 2000 34 511 523
5. Covariate imbalance and adjustment for logistic regression analysis of clinical trial data. Ciolino JD, Martin RH, Zhao W, Jauch EC, Hill MD, Palesch YY, J Biopharm Stat 2013 23 1383 1402 10.1080/10543406.2013.834912 24138438
6. Piantadosi S, Clinical Trials: a Methodologic Perspective New York: Wiley 2 2005
7. Response. Kernan WN, Makuch RM, J Clin Epidemiol 2001 54 105 10.1016/S0895-4356(00)00285-7
8. The method of minimization for allocation to clinical trials: a review. Scott NW, McPherson GC, Ramsay CR, Campbell MK, Control Clin Trials 2002 23 662 674 10.1016/S0197-2456(02)00242-8 12505244
9. Statistical comparison of random allocation methods in cancer clinical trials. Hagino A, Hamada C, Yoshimura I, Ohashi Y, Sakamoto J, Nakazato H, Control Clin Trials 2004 25 572 584 10.1016/j.cct.2004.08.004 15588744
10. Faulty assumptions in Atkinson's criteria for clinical trial design. Taves DR, J R Stat Soc 2004 167 179 181 10.1046/j.0964 - 1998.2003.00741.x
11. Handling covariates in the design of clinical trials. Rosenberger WF, Sverdlov O, Stat Sci 2008 23 404 419 10.1214/08-STS269
12. Repeated measures in clinical trials: analysis using mean summary statistics and its implications for design. Frison L, Pocock SJ, Stat Med 1992 11 1685 1704 10.1002/sim.4780111304 1485053
13. Randomized controlled trials with time-to-event outcomes: how much does prespecified covariate adjustment increase power? Hernández AV, Eijkemans MJ, Steyerberg EW, Ann Epidemiol 2006 16 41 48 10.1016/j.annepidem.2005.09.007 16275011
14. ANCOVA versus change from baseline had more power in randomized studies and more bias in nonrandomized studies. Van Breukelen GJP, J Clin Epidemiol 2006 59 920 925 10.1016/j.jclinepi.2006.02.007 16895814
15. Are unadjusted analyses of clinical trials inappropriately biased toward the null? Kent DM, Trikalinos TA, Hill MD, Stroke 2009 40 672 673 10.1161/STROKEAHA.108.532051 19164784
16. Measuring continuous baseline covariate imbalances in clinical trial data. Ciolino JD, Martin RH, Zhao W, Hill MD, Jauch EC, Palesch YY, Stat Methods Med Res 2011 doi:10.1177/0962280211416038
17. A substantial and confusing variation exists in handling of baseline covariates in randomized controlled trials: a review of trials published in leading medical journals. Austin PC, Manca A, Zwarenstein M, Juurlink DN, Stanbrook MB, J Clin Epidemiol 2010 63 142 153 10.1016/j.jclinepi.2009.06.002 19716262
18. The use of percentage change from baseline as an outcome in a controlled trial is statistically inefficient: a simulation study. Vickers AJ, BMC Med Res Methodol 2001 1 6 10.1186/1471-2288-1-6 11459516
19. Matthews JNS, Introduction to Randomized Controlled Clinical Trials Boca Raton, FL: Chapman & Hall/CRC 2 2006
20. Huitema B, The Analysis of Covariance and Alternatives: Statistical Methods for Experiments, Quasi-Experiments, and Single-Case Studies Hoboken, NJ: Wiley 2 2011
21. Beneficial effect of azathioprine and prediction of prognosis in primary biliary cirrhosis. Final results of an international trial. Christensen E, Neuberger J, Crowe J, Altman DG, Popper H, Portmann B, Doniach D, Ranek L, Tygstrup N, Williams R, Gastroenterology 1985 89 1084 1091 3899841
22. Choosing covariates in the analysis of clinical trials. Beach ML, Meier P, Control Clin Trials 1989 10 4 Suppl 161S 175S 2605965
23. Clinical trials in acute myocardial infarction: should we adjust for baseline characteristics? Steyerberg EW, Bossuyt PMM, Lee KL, Am Heart J 2000 139 745 751 10783203
24. Analysing controlled trials with baseline and follow up measurements. Vickers AJ, Altman DG, BMJ 2001 323 1123 1124 10.1136/bmj.323.7321.1123 11701584
25. Baseline comparisons in randomised clinical trials. Senn SJ, Stat Med 1991 10 1157 1160 1876802
26. Directional baseline differences and Type I error probabilities in randomized clinical trials. Overall JE, Magee KN, J Biopharm Stat 1992 2 189 203 10.1080/10543409208835039 1300213
27. Implications of chance baseline differences in repeated measurement designs. Overall JE, Doyle SR, J Biopharm Stat 1994 4 199 216 10.1080/10543409408835083 7951275
28. Assessment and implication of prognostic imbalance in randomized controlled trials with a binary outcome-a simulation study. Chu R, Walter SD, Guyatt G, Devereaux PJ, Walsh M, Thorlund K, Thabane L, PLoS One 2012 7 36677 10.1371/journal.pone.0036677 22629322
29. Subgroup analysis, covariate adjustment and baseline comparisons in clinical trial reporting: current practice and problems. Pocock SJ, Assmann SE, Enos LE, Kasten LE, Stat Med 2002 21 2917 2930 10.1002/sim.1296 12325108
30. Cohen J, Statistical Power Analysis for the Behavioral Sciences Hillsdale NJ: Lawrence Erlbaum 2 1988
31. Statistical power for analyses of changes in randomized controlled trials. Tu YK, Blance A, Clerehugh V, Gilthorpe MS, J Dent Res 2005 84 283 287 10.1177/154405910508400315 15723872
32. Analysis of data with imbalance in the baseline outcome variable for randomized clinical trials. Wei L, Zhang J, Drug Inf J 2001 35 1201 1214 10.1177/009286150103500417
33. Uses and abuses of analysis of covariance in clinical trials. Egger MJ, Coleman ML, Ward JR, Reading JC, Williams HJ, Control Clin Trials 1985 6 12 24 10.1016/0197-2456(85)90093-5 3987300
34. Evaluation of the result of a randomized controlled trial: how to define changes between baseline and follow up. Twisk J, Proper K, J Clin Epidemiol 2004 57 223 228 10.1016/j.jclinepi.2003.07.009 15066681
35. Subgroup analysis and other (mis)uses of baseline data in clinical trials. Assmann SF, Pocock SJ, Enos LE, Kasten LE, Lancet 2000 355 1064 1069 10.1016/S0140-6736(00)02039-0 10744093
36. Statistical methods for the identification and use of prognostic factors. Armitage P, Gehan EA, Int J Cancer 1974 13 16 36 10.1002/ijc.2910130104 4594339
37. Covariate imbalance and random allocation in clinical trials. Senn SJ, Stat Med 1989 8 467 475 10.1002/sim.4780080410 2727470
38. Testing for baseline balance in clinical trials. Senn SJ, Stat Med 1994 13 1715 1726 10.1002/sim.4780131703 7997705
39. How to select covariates to include in the analysis of a clinical trial. Raab GM, Day S, Sales J, Control Clin Trials 2000 21 330 342 10.1016/S0197-2456(00)00061-1 10913808
40. DHHS, Guidance for Industry E9: Statistical Principles for Clinical Trials Rockville MD: Department of Health and Human Services 1998
41. SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials. Chan A-W, Tetzlaff JM, Gøtzsche PC, Altman DG, Mann H, Berlin JA, Dickersin K, Hróbjartsson A, Schulz KF, Parulekar WR, Krleža-Jeric K, Laupacis A, Moher D, BMJ 2013 346 7586 10.1136/bmj.e7586 23303884
42. Quantifying the cost in power of ignoring continuous covariates imbalances in clinical trial randomization. Ciolino J, Zhao W, Martin R, Palesch Y, Contemp Clin Trials 2011 32 250 259 10.1016/j.cct.2010.11.005 21078415
43. Stratified randomization for clinical trials. Kernan WN, Viscoli CM, Makuch RW, Brass LM, Horwitz RI, J Clin Epidemiol 1999 52 19 26 10.1016/S0895-4356(98)00138-3 9973070
44. Reporting and analysis of trials using stratified randomisation in leading medical journals: review and reanalysis. Kahan BC, Morris TP, BMJ 2012 345 5840 10.1136/bmj.e5840 22983531
45. Baseline corrections in experimental and quasi-experimental clinical trials. Overall JE, Ashby B, Neuropsychopharmacology 1991 4 273 281 1867736
46. Covariate adjustment in randomized controlled trials with dichotomous outcomes increases statistical power and reduces sample size requirements. Hernández AV, Steyerberg EW, Habbema JD, J Clin Epidemiol 2004 57 454 460 10.1016/j.jclinepi.2003.09.014 15196615